Pathogensicherheit von ATMP
Biotechnologie
Abstract
Pathogen Safety of ATMP
New concepts of new treatments and highly innovative medicinal products, based on the improved understanding of the pathophysiology of diseases and the rapid development in the field of biology, biotechnology and medicine result in a purposive treatment of a range of acquired or inherited diseases for which there is previously an unmet medical need. These so-called Advanced Therapy Medicinal Products (ATMP) consist of gene therapy products, products containing viable cells and biotechnologically modified tissues. As for all biologicals there is a risk of transmission of pathogens to recipients also via these products. Measures to minimise these infection risks by the manufacturing process of most ATMP are commonly impossible to apply; during the production of cell and tissue products complex purification and, especially, pathogen inactivation steps cannot be implemented to inactivate and/or remove contaminants effectively. In contrast to ATMP, in the manufacturing process of classical biotherapeutics as plasma derived products, recombinant proteins and monoclonal antibodies dedicated manufacturing steps to inactivate and/or remove pathogens, especially human pathogenic viruses, are integrated. Essential measures for risk minimisation in ATMP include, therefore, intensive testing for contaminations of all raw and starting materials and the use of only non-reactive materials as well as the application of virus inactivation steps for potentially virus contaminated raw materials.
Zusammenfassung
Neue Therapiekonzepte, die auf einem verbesserten Verständnis der zugrunde liegenden Pathophysiologie von Krankheiten und dem Fortschritt in der Entwicklung der Biotechnologie beruhen, ermöglichen eine zielgerichtetere Behandlung verschiedener erworbener und angeborener Krankheiten. Zu diesen sog. Arzneimitteln für neuartige Therapien (Advanced Therapy Medicinal Products, ATMP) zählen sowohl Gentherapeutika als auch die i. d. R. mit aufwendigen Techniken hergestellten somatischen Zelltherapeutika und biotechnologisch bearbeiteten Gewebeprodukte. Wie alle Biologika bergen auch diese
